Rare disease gene therapy gains Prime status; FDA approves first drug for inflammatory arthritis nr-axSpA

→ Pittsburgh-based Krystal Biotech $KRYS has won the EMA’s Prime status for its gene therapy to treat dystrophic epidermolysis bullosa. The company says regulators were won over by their ongoing GEM-1 Phase 1/2 study coupled with non-clinical data.

→ The FDA on Thursday approved the first treatment for nr-axSpA, a type of arthritis that causes inflammation in the spine. The injection, called Cimzia, has been developed by UCB and was originally approved in 2008 and is currently also sanctioned for use in Crohn’s disease, rheumatoid arthritis, active ankylosing spondylitis (AS) and plaque psoriasis. The drug carries a boxed warning for increased risk of serious infections leading to hospitalization or death, including tuberculosis, bacterial sepsis, invasive fungal infections and others.

→ Dutch biotech Micreos wants to upend the way we deal with bacteria. Unlike traditional antibiotics, its endolysin technology is designed to target only unwanted bacteria while preserving the microbiome, to snuff out antibiotic resistant strains of bacteria and is engineered such that “resistance against endolysins is not expected.” To fuel its drug development, and aid the launch of its OTC eczema product, the Netherlands-based firm has secured €30 million (about $33.7 million) in funding.

With contribution by Natalie Grover.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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